Baxalta Incorporated, a global biopharmaceutical leader, announced that it has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for approval of Adynovi, an extended circulating half-life recombinant Factor VIII (rFVIII) treatment, for paediatric, adolescent and adult patients with haemophilia A and for use during surgery.
Currently licensed in the US as Adynovate [Antihemophilic Factor (Recombinant), PEGylated] and under regulatory review in Japan, Canada and Switzerland, Adynovi is the only rFVIII treatment for haemophilia A developed based on the demonstrated efficacy of Advate [Antihemophilic Factor (Recombinant)].
''We are dedicated to bringing Adynovi to haemophilia A patients all over the world,'' said Brian Goff, executive vice president and president, Hematology, Baxalta. ''As we build on our market-leading portfolio of direct factor replacement treatments, we continually bring new options to haemophilia patients, empowering them to manage their hemophilia, with their caregivers, in the way that works best for them.''
Adynovi was studied in patients, 12 to 65 years of age, in a prospective, global, multi-center, open label, non-randomized study; in patients 12 years of age and younger, in a prospective, uncontrolled, open-label, multi-center study; and for the perioperative control of hemostasis among 15 patients with severe hemophilia A undergoing surgical procedures. The EMA filing is based on data from these three phase 3 clinical trials, which Baxalta shared publicly in December 2015.
Haemophilia A is a challenging chronic disease; treatment regimens require regular infusions to reduce the risk of bleeding. Working closely with their health professionals, many patients continue to seek treatment options that can be better personalized to fit their needs, providing both effective bleed protection and simplified dosing schedules. Today, haemophilia affects more than 400,000 people globally and an estimated 75 per cent of people with haemophilia are inadequately treated. Baxalta continues to innovate in order to address some of the greatest challenges associated with hematologic disorders, including hemophilia.
Adynovate is built on the full-length Advate molecule, a leading treatment for haemophilia A that been used by patients worldwide for more than 12 years. Through a collaboration with Nektar Therapeutics, Adynovate leverages proprietary PEGylation technology designed to extend the amount of FVIII available for use in the body. The technology was selected because it maintains the integrity of the parent molecule (Advate) and reduces the time at which the body clears Adynovate, resulting in an extended circulating half-life. This proprietary technology has been used for more than 15 years in a number of approved medicines that treat chronic or serious conditions.
Common adverse reactions (=1 per cent of subjects) reported in the clinical studies were headache and nausea.
Advate has a demonstrated efficacy and safety profile for the treatment of haemophilia A. Advate is a full-length (derived from the complete FVIII gene) recombinant FVIII product that is processed without any blood-based additives. Because no blood-derived components are added at any stage of the manufacturing process, the potential risk of transmitting pathogens that may be carried in blood-based additives is virtually eliminated. There have been no confirmed reports of transmission of HIV, HBV or HCV with rFVIII treatments.
Advate is the world's most prescribed FVIII treatment. It is currently approved in 67 countries worldwide, including the United States, Canada, 28 countries in the European Union, Algeria, Argentina, Australia, Brazil, Brunei, Chile, China, Colombia, Ecuador, Hong Kong, Iceland, India, Iraq, Israel, Japan, Kuwait, Macau, Malaysia, Mexico, Morocco, New Zealand, Norway, Panama, Puerto Rico, Qatar, Russia, Saudi Arabia, Serbia, Singapore, South Korea, Suriname, Switzerland, Taiwan, Tunisia, Turkey, Ukraine, Uruguay, and Venezuela.
Serious adverse reactions seen with Advate are hypersensitivity reactions, including anaphylaxis, and the development of high-titer inhibitors necessitating alternative treatments to factor VIII.